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Today, the industry media STAT released a report that sorted out the important events expected to obtain clinical trial results or achieve regulatory progress in the first quarter of this year, including 11 innovative therapies across different fields, which may affect the direction of the biomedical industry in the new year.
KarXT（Karuna Therapeutics）
KarXT（Karuna Therapeutics）KarXT is developed by Karuna
Therapeutics develops innovative drugs
for the treatment of schizophrenia in adults.
It is a new drug
under development, oral and has the potential to become the first non-targeted dopaminergic and serotonergic pathway with dual mechanism.
KarXT consists of xanomeline and trospium
Chloride is composed of two active ingredients designed to activate muscarinic acetylcholine receptors in the brain while reducing the effect of
peripheral muscarinic acetylcholine receptors.
In August last year, Karuna announced that KarXT achieved its primary endpoint
in the EMERGENT-2 Phase 3 trial.
Karuna expects to announce the results of the second Phase 3 clinical trial of KarXT in the
first quarter of this year.
Karuna plans to submit a New Drug Application (NDA)
to the U.
S.
FDA in mid-2023.
If approved, it could be the first new class of drugs
to treat schizophrenia in 50 years.
with a dual mechanism that does not target the dopaminergic and serotonergic pathways.
Karuna expects to announce the results of the second Phase 3 clinical trial of KarXT in the
first quarter of this year.
If approved, it could be the first new class of drugs
to treat schizophrenia in 50 years.
Tovorafenib（Day One Pharmaceuticals）
Tovorafenib（Day One Pharmaceuticals）Tovorafinib is a highly specific pan-RAF kinase inhibitor that inhibits carrying BRAF fusion, or BRAF
The growth of V600 mutated tumors and brain permeability
.
Tovorafenib received breakthrough therapy designation and rare pediatric disease designation from the U.
S.
FDA for the treatment of pediatric low-grade gliomas (pLGGs)
carrying activated RAF variants.
In June last year, tovorafenib achieved positive results
in a pivotal Phase 2 clinical trial for the treatment of pLGG patients.
Of the first 22 patients that could be evaluated, tovorafinib achieved an overall response rate (ORR)
of 64%.
The final results of this FIREFLY-1 pivotal Phase 2 trial, which includes all participants, will be announced
in the first quarter of this year.
.
The final results of this FIREFLY-1 pivotal Phase 2 trial, which includes all participants, will be announced
in the first quarter of this year.
Nexletol（Esperion Therapeutics）
Nexletol（Esperion Therapeutics）Nexlizet（bempedoic
acid/ezetimibe) is a non-statin oral cholesterol-lowering combination therapy, a "first-in-class" ATP citrate lyase (ACL) inhibitor that lowers cholesterol biosynthesis and thereby LDL-C levels
.
This therapy was approved by the FDA in 2020 for the treatment of heterozygous familial hypercholesterolemia (HeFH) and atherosclerotic cardiovascular disease (ASCVD
) in adults.
In December, Esperion announced that Nexlizet had achieved its primary endpoint in a Phase 3 clinical trial that significantly reduced the risk of patients developing serious adverse cardiovascular disease, and full trial results are expected to be presented
at the American College of Cardiology (ACC) meeting in March.
.
Esperion announced that Nexlizet achieved its primary endpoint in a Phase 3 clinical trial that significantly reduces the risk of patients developing serious adverse cardiovascular disease, and full trial results are expected to be presented
at the American College of Cardiatrics (ACC) meeting in March.
Elahere（ImmunoGen）
Elahere（ImmunoGen）Elahere（mirvetuximab
soravtansine) is a "first-in-class" antibody conjugate (ADC)
targeting folate receptor α (FRα).
Elahere received accelerated FDA approval in November last year as a monotherapy for the treatment of treated patients with advanced ovarian cancer with high FRα expression and resistance to platinum-containing therapy
.
The approval is based on the results of the pivotal Phase 3 clinical trial, SORAYA, which showed that the trial met the primary endpoint and the objective response rate (ORR) confirmed by investigator evaluation was 31.
7% (95%
CI: 22.
9%, 41.
6%), including 5 complete responses (CR), independent of
previous treatment lines or prior PARP inhibitor (PARPi) therapy.
The results of the confirmatory testing required for final marketing approval by Elahere are expected to be available
in the first quarter of this year.
In October 2020, Huadong Pharmaceutical obtained the exclusive development and commercialization rights
of the drug in Greater China for more than US$300 million.
) targeting folate receptor α (FRα).
The results of the confirmatory testing required for final marketing approval by Elahere are expected to be available
in the first quarter of this year.
Vyvgart（Argenx）
Vyvgart（Argenx）Vyvgart (efgartigimod) is a "first-in-class" therapy
that targets the Fc receptor (FcRn).
The therapy was approved in December 2021 for the treatment of generalized myasthenia gravis (gMG), becoming the first FDA-approved FcRn blocker
.
Vyvgart reduces pathogenic IgG antibodies and blocks the IgG recycling process
.
The role of the FcRn receptor is to prevent the degradation of IgG, so by preventing the binding of IgG to FcRn, it can lead to faster depletion of IgG antibodies mediating autoimmune diseases, thereby reducing disease symptoms
.
The top-line results of Vyvgart's trial for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) are expected this year
.
targeting the Fc receptor (FcRn).
The therapy was approved in December 2021 for the treatment of generalized myasthenia gravis (gMG), becoming the first FDA-approved FcRn blocker
.
The top-line results of Vyvgart's trial for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) are expected this year
.
Lovo-cel（bluebird bio）
Lovo-cel（bluebird bio）lovotibeglogene
Autotemcel (lovo-cel) is a one-time gene therapy for sickle cell disease (SCD), which aims to add a functional β globulin gene (βA-T87Q globulin) to the hematopoietic stem cells of SCD patients, thereby reducing the level of hemoglobin S (HbS) and the production
of sickle red blood cells.
Lovo-cel has been granted RMAT designation by the FDA for Advanced Therapies in Regenerative Medicine (RMAT) and Rare Pediatric Disease (RPD).
A Phase 1/2 clinical trial and a Phase 3, long-term efficacy and safety trial are underway at Lovo-Cel, and the company expects to submit a biologics license application
in the first quarter of this year.
.
Sickle cell disease (The Company expects to file a biologics license application
in the first quarter of this year.
Roctavian（Biomarin Pharmaceuticals）
Roctavian（Biomarin Pharmaceuticals）Roctavian is a hemophilia A gene therapy
developed by BioMarin.
Roctavian uses an AAV5 viral vector to deliver transgenes expressing factor VIII, making it possible for patients to receive only a one-time treatment for hepatocytes to continue expressing factor VIII, eliminating the need for long-term prophylactic factor injections
.
The US FDA granted this gene therapy breakthrough therapy designation (BTD) and regenerative medicine advanced therapy designation
in 2017 and 2021, respectively.
The results of the three-year clinical trial data requested by the FDA for Roctavian will be released earlier this year, and the completion date for the FDA's review of Roctavian is March 31 this year
.
.
The results of the three-year clinical trial data requested by the FDA for Roctavian will be released earlier this year, and the completion date for the FDA's review of Roctavian is March 31 this year
.
Exa-cel（Vertex Pharmaceuticals & CRISPR Therapeutics）
Exa-cel（Vertex Pharmaceuticals & CRISPR Therapeutics）Exa-cel (formerly known as CTX001) is an investigational in vitro CRISPR/Cas9 gene editing therapy that produces high levels of fetal hemoglobin (HbF) in red blood cells by genetically editing a patient's own hematopoietic stem cells to produce high levels of fetal hemoglobin (HbF) in the treatment of sickle cell disease and transfusion-dependent β thalassemia (TDT).
。 At the American Society of Hematology (ASH) annual meeting in December, the two companies presented the latest trial results of exa-cel, which showed that of the 44 patients treated with exa-cel, 42 did not need to receive red blood cell transfusions
.
Among patients with SCD treated with exa-cel, none developed the vascular occlusive crisis (VOC)
common in SCD patients.
Among them, the patient with the longest follow-up time has been maintained for 32.
3 months without VOC
.
In September last year, exa-cel received FDA clearance to submit a rolling application for the treatment of SCD and TDT, and is expected to complete the Biologics License Application (BLA)
in the first quarter of this year.
The submission of the Biologics Licensing Application (BLA)
is expected to be completed in the first quarter of this year.
VERVE-101（Verve Therapeutics）
VERVE-101（Verve Therapeutics）VERVE-101 is a single-base editing therapy
for the treatment of patients with familial hypercholesterolemia (HeFH).
Last July, a volunteer in New Zealand received the therapy, making VERVE-101 the first single-base editing therapy
in vivo to enter clinical development.
This therapy inactivates PCSK9 by changing one letter of the PCSK9 gene in the
patient's cells.
PCSK9 is a popular target for reducing LDL-C, and its efficacy in inhibiting its activity has been verified
by a number of FDA-approved therapies.
In October last year, Verve submitted an IND application for VERVE-101 to the FDA, and in November it received notice from the FDA to suspend clinical trials
of VERVE-101.
Verve has received questions from the FDA regarding this trial and, based on its interactions with the FDA, expects to provide progress this year to address FDA concerns and initiate clinical trials
of this therapy in the United States as soon as possible.
for the treatment of patients with familial hypercholesterolemia (HeFH).
Verve has received questions from the FDA regarding this trial and, based on its interactions with the FDA, expects to provide progress this year to address FDA concerns and initiate clinical trials
of this therapy in the United States as soon as possible.
Fazirsiran (Takeda & Arrowhead Pharmaceuticals)
Fazirsiran (Takeda & Arrowhead Pharmaceuticals)Fazirsiran is a potential "first-in-class" RNAi therapy
for α-1 antitrypsin deficiency (AATD).
Data from a small open-label study trial published last June showed that 16 patients with liver disease associated with α-1 antitrypsin deficiency had a significant reduction in the accumulation of mutant AAT protein in the liver, which is one of
the typical features of the disease, after receiving fazirsiran.
While accepting 200
Of the 12 people treated with high-dose fazirsiran, 7 experienced resolution of liver fibrosis
.
The results of the study have been published in the New England Journal of Medicine
.
Arrowhead and Takeda expect to release top-line data from fazirsiran's Phase 2 trial in SEQUOIA in January, as well as plans for
related Phase 3 clinical trials.
for α-1 antitrypsin deficiency (AATD).
Top-line data from fazirsiran's Phase 2 trial in SEQUOIA and plans for
related Phase 3 clinical trials are expected to be released in January this year.
mRNA-1010（Moderna）
mRNA-1010（Moderna）mRNA-1010 is a quadrivalent seasonal influenza mRNA vaccine candidate that targets four seasonal influenza strains recommended by the World Health Organization (WHO), including influenza A strains H1N1 and H3N2, and influenza B strains Yamagata and Victoria
.
The vaccine is ongoing in a Phase 3 clinical trial in thousands of volunteers to compare the immunogenicity
of mRNA-1010 with an active control.
The findings, which are expected in the first half of next year, are intended to support initial regulatory approval
for mRNA-1010.
.
The findings, which are expected in the first half of next year, are intended to support initial regulatory approval
for mRNA-1010.
Resources:
[1] The biotech scorecard for the first quarter: 19 stock-moving events to
watch.
Retrieved January 3, 2023 from
Resources:
[1] The biotech scorecard for the first quarter: 19 stock-moving events to
watch.
Retrieved January 3, 2023 from
Resources:
Resources:
[1] The biotech scorecard for the first quarter: 19 stock-moving events to
watch.
Retrieved January 3, 2023 from
Retrieved January 3, 2023 from
